One Step Closer to a Cure for Leukemia without Chemotherapy or Radiation

At most hospitals, children with relapsed acute lymphoblastic leukemia (ALL) who aren’t responding well to chemotherapy would be running out of options. But Seattle Children Research Institute’s researchers are one step closer to finding a cure. Starting this month, patients who have relapsed ALL will have the option of participating in a new clinical trial if they are not responding to chemotherapy and have a less than 20 percent chance of survival.

Harnessing life-saving cells in patients’ blood

The new treatment—called cellular immunotherapy—involves drawing blood from the patient, reprogramming their infection-fighting T cells to find and destroy cancer cells, and infusing the blood back into their body.

Only three other institutions in the country are conducting this type of clinical trial, which involves using a specialized high-tech facility to manufacture the personalized therapy using each patient’s blood.

“The reprogrammed T cells are genetically modified to zero in on the cancer cells and attack only those cells just like they would get rid of a viral infection,” says Michael Jensen, MD, director of the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute. “It is our hope as we develop and refine this targeted form of immunotherapy we can become less reliant on chemotherapy and radiation therapy that often cause lifelong debilitating side effects.”

At Children’s, 86.9 percent of patients who are diagnosed with ALL for the first time survive —which is a higher survival rate than the national average of 85 percent. But if a patient goes through treatment and the cancer comes back, the odds are against them.

“Less than 20 percent of patients with relapsed ALL typically survive, so it’s imperative to find a cure,” says Rebecca Gardner, MD, the researcher leading the clinical trial at Seattle Children’s. “And ultimately we hope to apply this same type of therapy for patients with many different types of cancer.”

From laboratory testing to treatment for patients

One of the most challenging steps in biomedical research is to take a therapy from tests in the lab—no matter how thoroughly it has been tested—to the very first patients.

The main goals of this first phase of research in patients—known as a Phase I clinical trial—are to determine the dose of the T cells that is tolerable, to assess the safety of this type of treatment, and to study the way the T cells work in treated patients.

The first research subjects to try the new therapy will be 18- to 26-year-old patients. After that first phase, the trial will be open to patients age one to 26. Why start first with young adults? “In the last decade or so, there’s been a lot of interest in the Adolescent and Young Adult (AYA) population,” said Dr. Gardner. “Their leukemia is more similar to pediatric leukemia than adult leukemia, which typically presents in the elderly population.” The U.S. Food and Drug Administration, or FDA, also requires safety data first for adult patients before moving to the pediatric population.

Research participants will receive clinical care mainly in the outpatient Cancer and Blood Disorders Center at Seattle Children’s Hospital. Few institutions are prepared to provide care to patients undergoing this type of treatment given the sophisticated technology needed to develop the personalized cures and the potential for serious side effects.

“Children’s is one of only a few institutions that brings together the technology with the research and clinical expertise,” says Dr. Jim Hendricks, president of Seattle Children’s Research Institute. “These trials would never be possible without the dedication of our researchers, physicians, nurses and hospital community, including the donors who have made these clinical trials possible.”

Philanthropy’s role in this trial

The work being done in the lab and research-related patient care costs for this Phase 1 trial are being funded through philanthropy.

Our research efforts received a big boost in January 2012, thanks to a $5 million gift from Ben Towne Pediatric Cancer Research Foundation, spearheaded by Jeff and Carin Towne. Jeff and Carin’s son, Ben Towne, died at age three in 2008 after a two-year battle with neuroblastoma, a particularly aggressive form of pediatric cancer. The Towne’s generosity also helped establish the Ben Towne Center for Childhood Cancer Research.

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Seattle Children’s provides healthcare for the special needs of children regardless of race, color, creed, national origin, religion, sex (gender), sexual orientation or disability. Financial assistance for medically necessary services is based on family income and hospital resources and is provided to children under age 21 whose primary residence is in Washington, Alaska, Montana or Idaho.